DIY Gene Editing: Fast, Cheap—and Worrisome

Kian Sadeghi has postponed homework assignments, sports practice and all the other demands of being a 17-year-old high-school junior for today. On a Saturday afternoon, he is in a lab learning how to use Crispr-Cas9, a gene-editing technique that has electrified scientists around the world—and sparked a widespread debate about its use.

Scientific breakthroughs often raise big ethical questions. Moral concerns around the 1996 cloning of Dolly the sheep or the 2000 announcement of a rough draft of the human genome still reverberate today. The public benefits from scientific advances, particularly in improving health. But some scientists say the power to alter the DNA of plants, animals or people, and the profound impact such changes may have on individuals and society, merits public discussion.

Crispr gene editing by amateurs and hobbyists brings an unusual set of challenges. Crispr-Cas9 is easier, faster and cheaper than previous gene-editing techniques. While that raises the prospect of people with nefarious intent gaining access, the greater concern with amateur enthusiasts is that someone might make a seemingly innocuous gene edit in a fungus, insect or plant that turns out to wreak havoc on the environment.

“The question is, can we rely on individuals to conduct their experiments in an ethical and appropriately safe way?” says Maxwell Mehlman, a professor of law and bioethics at Case Western Reserve University, who is working with do-it-yourself scientists to develop DIY Crispr ethical guidelines. “The jury is out,” he says. “Crispr is too new. We have to wait and see.”

There are those who believe that given the importance of the ethical debate, the more people know about Crispr—including hands-on experience with it—the better. A do-it-yourself Crispr kit with enough material to perform five experiments gene-editing the bacteria included in the package is available online for $150. Genspace, the Brooklyn, N.Y., community lab where Mr. Sadeghi is learning how to use Crispr to edit a gene in brewer’s yeast, charges $400 for four intensive sessions. More than 80 people have taken the classes since the lab started offering them last year.

Both the kit and the lab allow people to work only on harmless organisms. And Genspace is an example of the kind of educational program that many researchers are racing to develop—not just explaining how to use the technology but also discussing when and how it should be used.

That discussion continues to bubble at the highest levels, as scientists wrestle with what limits should be imposed on Crispr inside their labs. A scientific advisory committee set up by the National Academy of Sciences and the National Academy of Medicine issued a report this month that supports human genome editing to try to treat and prevent diseases, but says more public discussion is needed for other uses, such as editing genes in eggs, sperm or embryos, which could be passed on to future offspring.

At the classroom level, Dana Waring Bateman, co-founder and education director at Harvard University’s Personal Genetics Education Project, helped create a Crispr lesson for high-school teachers. The lesson doesn’t involve doing an experiment. Rather, it covers the history of Crispr and gene editing, some of the key findings, and the moral questions Crispr raises.

Ms. Bateman, who visits schools to discuss the ethics of gene editing, says she asked a group of seventh-grade students whether Crispr should be deployed to bring extinct animals back to life. After a spirited discussion, one student asked, “How can we decide if we aren’t sure what will happen?” Ms. Bateman replied that such questions will increasingly be part of public debate, and that everyone, including 12-year-olds, can benefit from learning about Crispr.

At Genspace, Ellen Jorgensen, a molecular biologist and one of the lab’s founders, tells people in the workshop before they get started that working on a gene edit can help them better understand how to use Crispr, and when they shouldn’t. The lab takes safety precautions: Participants don’t work with pathogens, and as a general rule, they aren’t allowed to take genetically modified organisms or hazardous chemicals out of the lab. A small committee reviews requests by Genspace members who propose projects, and turns down those it considers ill-conceived or potentially dangerous.

In the workshop, if the participants correctly edit the gene in brewer’s yeast, the cells will turn red. In between the prep work, the classmates swap stories on why they are there. Many have personal Crispr projects in mind and want to learn the technique.

Kevin Wallenstein, a chemical engineer, takes a two-hour train ride to the lab from his home in Princeton, N.J. Crispr is a hobby for him, he says. He wants to eventually use it to edit a gene in an edible fruit that he prefers not to name, to restore it to its historical color. “I always wondered what it would look like,” he says.

At the workshop, Mr. Wallenstein shares his Crispr goal with Will Shindel, Genspace’s lab director. Mr. Shindel is enthusiastic; he has started his own Crispr project, a longtime dream to make a spicy tomato. Both men say they aren’t looking to commercialize their ideas—but they would like to eat what they create someday, if they get permission from the lab. “I’m doing it for fun,” Mr. Shindel says.

When Mr. Sadeghi first wanted to try Crispr, the teenager emailed 20 scientists asking if they would be willing to let him learn Crispr in their labs. Most didn’t respond; those that did turned him down. So he did a Google search and stumbled upon Genspace. When he shared the lead with his science teacher at the Berkeley Carroll School in Brooklyn, Essy Levy Sefchovich, she agreed to take the course with him.

When Mr. Shindel describes the steps of the experiment, Ms. Sefchovich takes notes. She is hoping to create a modified version of the yeast experiment so all her students can try Crispr in class.

Later, Mr. Sadeghi recounts that the hardest part of the day was handling the micropipette, the lab tool he used to mix small amounts of liquid. He says he still feels clumsy. Ms. Sefchovich reassures him he’ll get the hang of it; he just needs to practice.

“It’s like driving,” she tells him. “You learn the right feel.” Mr. Sadeghi doesn’t have his driver’s license yet. He figures he’ll do Crispr first.

More About

BY TOM IRELAND
THE GUARDIAN
Josiah Zayner, 36, recently made headlines by becoming the first person to use the revolutionary gene-editing tool Crispr to try to change their own genes. Part way through a talk on genetic engineering, Zayner pulled out a syringe apparently containing DNA and other chemicals designed to trigger a genetic change in his cells associated with dramatically increased muscle mass. He injected the DIY gene therapy into his left arm, live-streaming the procedure on the internet.
The former Nasa biochemist, based in California, has become a leading figure in the growing “biohacker” movement, which involves loose collectives of scientists, engineers, artists, designers, and activists experimenting with biotechnology outside of conventional institutions and laboratories.
Despite warnings from the US Food and Drug Administration (FDA) that selling gene therapy products without regulatory approval is illegal, Zayner sells kits that allow anyone to get started with basic genetic engineering techniques, and has published a free guide for others who want to take it further and experiment on themselves.
Was administering a dose of Crispr on yourself an experiment, or a stunt to show what amateur scientists/biohackers can do?
Both. The technical feasibility of what I did is not under question – researchers have done this many times, in all sorts of animals. But there’s a barrier – people are afraid of it, and just talk about the possibilities in humans. I wanted to break that down, to say “Hey look, the tools are inexpensive, and somebody with a bit of knowledge can actually go through with these experiments”.
I chose to start with the gene for myostatin [a protein that regulates muscle growth], because it has been extensively studied, and it produces an obvious change if it has worked.
So, how is your arm looking?
In similar experiments with animals, you only start to see results after four to six months of treatment. I would expect that the DNA in some of the cells of my arm has changed, but I am still working on developing assays [tests] to try and detect that. As to whether the actual size of the muscle changes, I’m more sceptical.
Changing the way one gene behaves can have a huge number of knock-on effects on the way other genes are regulated or expressed. Do you really know what you’re doing?
It’s a good question. These things are complicated, and obviously with things like this there are lots of unknowns. I look at what the possible negative outcomes are and ask: “Are those risks insignificant enough that I’m willing to undertake this experiment?” Based on the data I read, for a local injection the answer was yes. A treatment that blocks myostatin throughout the whole body? That would be much more hazardous – you would be messing with the muscles of your heart.
You support the idea of people attempting gene therapy and other experimental procedures on themselves. What’s wrong with the existing system, where treatments are thoroughly tested by professionals before being approved for use?
If we’re going to do these experiments you have to balance two things: how many people can possibly die from testing their own products or making them available prematurely, versus how many people have genetic disorders and are just dying because they don’t have access to them. I think there’s a huge imbalance, where we’re overprotective of hurting people instead of offering a chance to millions of people who are dying right now.
As human beings we’re very big on freedoms, equality, equal rights. What’s more of an equal right than being able to control what genes we have? I think people should be able to choose that. I’m not saying anything I can do can help treat people, but treating things genetically is the ultimate medicine.
I grew up in the 90s with the computer hacker movement, the development of the internet – the whole open-source movement was amazing. Who created Linux, the most used operating system ever? Not students from Harvard or Cambridge, but Linus Torvalds, a student in Finland working in his apartment.
I don’t think for a second I’m going to be the mastermind behind a great biotech revolution, but I think there’s some brilliant person waiting to be discovered out there that could be.

genomic privacy

BY CURTIS AND HEREWARD
THE CONVERSATION
We are approaching a time when you might be too scared to have your genome sequenced. Only last week, a US senator called for an investigation into the privacy policies of direct-to-consumer DNA companies.

There is something like gold flowing through the veins of 100-year-old Maria Tegas, and everyone wants a piece of the treasure. The centenarian, who lives in a poor and remote area of central eastern Sardinia – in one of 14 villages known to geneticists and genealogists as the Blue Zone – has not had an easy life.

professional regulation

BY TOM IRELAND
THE GUARDIAN
Josiah Zayner, 36, recently made headlines by becoming the first person to use the revolutionary gene-editing tool Crispr to try to change their own genes. Part way through a talk on genetic engineering, Zayner pulled out a syringe apparently containing DNA and other chemicals designed to trigger a genetic change in his cells associated with dramatically increased muscle mass.

BY AMY DOCKSER MARCUS
WALL STREET JOURNAL
Kian Sadeghi has postponed homework assignments, sports practice and all the other demands of being a 17-year-old high-school junior for today. On a Saturday afternoon, he is in a lab learning how to use Crispr-Cas9, a gene-editing technique that has electrified scientists around the world—and sparked a widespread debate about its use.

emerging technologies

BY LAURIE MCGINLEY
WASHINGTON POST
In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease. The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for hemophilia, sickle-cell anemia and an array of other genetic diseases. Yet the products, should they reach patients, are likely to carry stratospheric prices — a prospect already worrying consumer advocates and economists.

The Associated Press
CBC.ca
Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease. The experiment was done Monday in California on 44-year-old Brian Madeux.

gene patents

BY ANDRÉ PICARD
THE GLOBE AND MAIL
‘Gene patents no longer need to stand in the way of diagnosing life-threatening disease.”
That’s how Alex Munter, president and chief executive officer of the Children’s Hospital of Eastern Ontario, summed up the impact of an out-of-court settlement in the lawsuit CHEO launched against Transgenomic Inc. in 2014. Transgenomic, a biotechnology company based in Omaha, Neb., owns five gene patents related to the potentially deadly heart condition Long QT syndrome.

BY MICHAEL SLEZAK
NEWSCIENTIST
Your genes are no longer patentable in Australia. The country’s highest court found unanimously that two previous Australian judgments allowing patents of genes were wrong, and they do not constitute a patentable invention.

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BY KELLY SERVICK
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